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• W4240678632 abstract "In CHERISH, a Phase 3, randomized, double-blind, sham-controlled study (NCT02292537), nusinersen demonstrated significant and clinically meaningful improvement in motor function vs sham control in children with later-onset SMA. Within CHERISH, nusinersen was associated with reduced caregiver impact and less decline in HRQoL vs sham control. Children who completed CHERISH were eligible to enrol in the SHINE open-label extension study (NCT02594124); all participants receive nusinersen 12 mg every 4 months after a protocol amendment. SHINE interim data from 27 August 2019 were evaluated. ACEND and PedsQL Generic Core and Neuromuscular Module (NM) were administered to caregivers of children in CHERISH and SHINE to assess the effects of longer-term treatment with nusinersen on caregiver impact and HRQoL. Higher scores (range 0–100) in the ACEND and PedsQL scales indicate reduced caregiver impact and improved HRQoL. ACEND scores among caregivers improved (mean change [95% CI] from baseline of CHERISH to Day 1170) for the nusinersen-treated group in CHERISH/SHINE (n=67) in 5/7 subdomains: Feeding/Grooming/Dressing: 10.3 (5.99,14.54), Sitting/Playing: 1.1 (-2.18,4.45), Transfers: 3.5 (-0.68,7.70), Mobility: 5.0 (-0.47,10.45), and Finance: 0.7 (-3.76,5.25). Those treated with nusinersen in CHERISH/SHINE who were younger at first dose (≥2–<3.5 y, and ≥3.5–<5 y) showed greater benefits in caregiver impact than older children (≥5–<9.5 y) in 6/7 ACEND subdomains. PedsQL generic total score improved from baseline of CHERISH to Day 1170 for children treated with nusinersen in CHERISH/SHINE (n=63): mean (95% CI) change was 2.3 (-1.08,5.72); whereas the PedsQL NM score stabilized over time: -0.2 (-3.79,3.49). In addition to significant and clinically meaningful improvements in motor function, nusinersen was associated with reduced impact for caregivers and improvements in HRQoL for children with later-onset SMA over 3 years as measured by the ACEND and PedsQL, respectively." @default.
• W4240678632 created "2022-05-12" @default.
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• W4240678632 date "2020-10-01" @default.
• W4240678632 modified "2023-10-16" @default.
• W4240678632 title "SMA – THERAPY" @default.
• W4240678632 doi "https://doi.org/10.1016/j.nmd.2020.08.268" @default.
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